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As research continues to evolve, so does our understanding of beta (β)-thalassemia, and new treatment options are being developed.

Prenatal Screening and Genetic Counseling

Because beta-thalassemia is a Genetic diseasea disease that is caused by a genetic change, it can be identified and diagnosed through prenatal screening. This can provide early diagnosis and allow people to better understand how beta-thalassemia can be managed. They may also be recommended to a genetics counselor who can help screen other family members and identify a match for a stem cell transplant if needed. 

Gene Therapy

Similar to a donor stem cell (or allogeneic) transplant, gene therapy is a treatment option that has the potential to treat beta-thalassemia in some patients. Unlike a stem cell transplant, however, gene therapy uses a patient's own cells. This type of therapy has the potential to treat the disease at the genetic level.

There are 2 main types of gene therapy: gene addition and gene editing.


In gene addition, copies of functional genes are added to a cell to help do the work of a defective gene. The addition of functional genes can take place either inside (in vivo) or outside of the body (ex vivo).


In gene editing, targeted breaks are created in DNA, with or without instructions for how to repair the break. The goal of these breaks is to disrupt or correct the function of a gene.

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Take a closer look at how gene therapy works at genehome, an educational resource for beginners to advanced learners.

Caregiver Tip

At your next annual comprehensive care visit, consider asking if you or your loved one is currently eligible for any advanced treatment options.

Keep the Conversations Going

As the understanding of beta-thalassemia evolves and new treatment options are developed, staying informed can give you the information you need to have more meaningful conversations with your doctor about living with beta-thalassemia, your long-term goals, and evolving options.